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Researchers at Vor Bio are conducting a global phase 3 clinical study (UPSTREAM MG) for adults with generalized myasthenia gravis (gMG) who are AChR or MuSK+. The study will evaluate whether treatment with the investigational drug telitacicept can reduce the level of disease-causing B cells and autoantibodies, and improve muscle weakness and fatigue, in people…

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No treatments are currently approved and available for limb-girdle muscular dystrophy type 2i (LGMD2i) in the U.S., but that may change later this year. The U.S. Food and Drug Administration (FDA) has accepted an application from Bridgebio Pharma seeking approval for its experimental oral therapy BBP-418. The agency granted the application priority review, reducing the…

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The MDA Quest Media Product Guide is designed to help you find the products you need to live a more independent, stylish, fun, and all-around great life. All the products you see here were chosen by MDA Ambassadors, who shared exactly how each product helps them in their daily lives. Find something for yourself, or…

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During a recent facioscapulohumeral muscular dystrophy (FSHD) wellness group meeting via Zoom, a friend of mine shared a poem by May Sarton that prompted the question: “Do I think of my body as an old friend or as an enemy?” I realized that my attitude toward my body has grown worse with recent difficulties. I…

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As someone living with spinal muscular atrophy (SMA), I know how important it is for children with disabilities to have full access to education. With accommodations for my mobility, I took honors and AP classes in high school, then went on to earn a master’s degree at Emerson College. I’m using my education to pursue…

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Neuromuscular diseases can impact muscles throughout the body, including the heart. Because heart health is essential to overall health, researchers are working to understand how different neuromuscular diseases affect the heart and why effects can vary between people with the same diagnosis. There are still open questions, but over the last decade, scientists have learned…

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For many people living with neuromuscular diseases — or any disability — the desire to become a parent is steady and deeply felt, even when the path looks different from what others might expect. Pregnancy may be one option, but it is far from the only one. Foster care, adoption (domestic or international), and surrogacy…

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Fiona Cauley, 29, brings attention to Friedreich ataxia (FRDA or FA) every time she sits on a stage. A comedian living with FRDA, Fiona candidly shares her observations about living with a disability. Before her diagnosis, Fiona played volleyball and soccer. When her first symptoms appeared in high school — loss of balance and slurred…

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Access to durable medical equipment (DME) is vital for safety, mobility, and greater independence. For Chuck Vrasich, of Illinois, a light, portable lift lets him move around his home without worrying about falls. For Jo Ann S. Rincon, of Texas, a toilet lift provides the support she needs to use the bathroom independently. Both of…

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Like many expectant moms, Savannah Jordan of California experienced a mix of excitement, nerves, and uncertainty during her first pregnancy, in 2018. But living with limb-girdle muscular dystrophy (LGMD) added another layer of questions. Jessery Picard Would her neuromuscular disease affect the pregnancy? Would pregnancy worsen her symptoms? Should she anticipate complications? What would the…

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